ARGININE SUPPLEMENTATION IN SICKLE CELL ANEMIA-PHYSIOLOGICAL AND PROPHYLATIC

镰状细胞贫血症中精氨酸的补充——生理学和预防学

• 批准号: 7379517
• 负责人: Lillian E McMahon
• 金额: $ 0.69万
• 依托单位: BOSTON UNIVERSITY MEDICAL CAMPUS
• 依托单位国家: 美国
• 财政年份: 2005
• 资助国家: 美国
• 起止时间: 2005-12-01 至 2006-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7379517
• 关键词: ARGININE; SUPPLEMENTATION; SICKLE; CELL; ANEMIA

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Sickle cell disease is a serious inherited disease of the red blood cells resultiing in sever anemia and episodes of occlusion of the blood vessels. This vaso-occlusion affects every organ system in the body and results in recurrent episodes of pain, overwhelming infections and progressive organ damage. Nitric oxide is a substance produced by arginine and has many functions that could impact favorably in vaso-occlusion in sickle cell disease. Arginine is an aminoacid that has been shown to raise nitric oxide and have benificial results in subjects with sickle cell disease, and will help to determine if daily arginine is a benificial investigational drug for subjects with sickle cell disease. This study is a double-blind, placebo-controlled trial in which eligible subjects (children, adolescents, and adults) with sickle cell disease will receive either arginine or placebo by mouth during 12 weeks. Subjects will be randomized to recieve placebo or one of two doses of arginine (0.04g/kg/day).

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得主要资金，因此可以在其他CRISP条目中表示。所列机构为中心，不一定是研究者所在机构。镰状细胞病是一种严重的遗传性红细胞疾病，可导致严重贫血和血管闭塞。这种血管阻塞影响身体的每个器官系统，并导致疼痛的反复发作，压倒性的感染和进行性器官损伤。一氧化氮是由精氨酸产生的物质，具有许多功能，可以有利地影响镰状细胞病的血管闭塞。精氨酸是一种氨基酸，已被证明可提高一氧化氮水平，对镰状细胞病受试者有益，并将有助于确定每日精氨酸是否是镰状细胞病受试者的有益研究药物。本研究是一项双盲、安慰剂对照试验，符合条件的镰状细胞病受试者（儿童、青少年和成人）将在12周内口服精氨酸或安慰剂。受试者将随机接受安慰剂或两种剂量的精氨酸（0.04 g/kg/天）之一。